Gene Transfer Clinical Trial for Krabbe Disease (NCT04693598) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Gene Transfer Clinical Trial for Krabbe Disease
United States6 participantsStarted 2021-11-05
Plain-language summary
This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.
Who can participate
Age range1 Day – 12 Months
SexALL
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Inclusion criteria
✓. Diagnosis of infantile Krabbe disease, characterized by the following criteria outlined below:
✓. Age at the time of screening: 1 day to 12 months
✓. Participant has been deemed eligible for treatment with HSCT (standard of care) and a fully myeloablative reduced intensity/toxicity conditioning regimen (RIC/RTC) is/has been used
✓. Participant's parents or legal guardian consents to participate in the study and provides informed consent according to IRB guidelines prior to any study procedures being performed
✓. Parent(s) and/or legal guardian able to comply with the clinical protocol
✓. Participant must have adequate organ function at time of screening as measured by:
Exclusion criteria
✕. History of prior treatment with a gene therapy product
✕. Presence of major congenital anomaly or any other condition that affects neurodevelopmental function
✕. Presence of any neurocognitive deficit or brain damage not attributable to Krabbe disease
✕. Active aspiration
✕. Signs of active infection or disease from cytomegalovirus, adenovirus or other viruses
✕. HIV positive
What they're measuring
1
Safety as assessed by incidence and severity of adverse events and serious adverse events that are attributed to FBX-101.
Timeframe: 24 months
2
Safety as assessed by HSCT incident of engraftment.